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Connection

Terence Flotte to Humans

This is a "connection" page, showing publications Terence Flotte has written about Humans.
Connection Strength

2.827
  1. Eichler F, Cataltepe OI, Daci R, Puri AS, Taghian T, Jiang X, Shazeeb MS, Kuhn A, Hader A, Celik H, Vardar Z, Lewis CJ, Artinian R, Nagy A, Vachha B, Thompson R, Gallagher T, Bateman S, Parzych J, Spanakis SG, Vaughn TA, Pier K, De Boever E, Abbott MA, D Ambrosio E, Kokoski D, Blackwood M, Drummond E, Ratai EM, Townsend EL, McLaughlin H, Tifft CJ, Keeler AM, Sena-Esteves M, Gray-Edwards HL, Flotte TR. Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial. Nat Med. 2025 Sep; 31(9):2927-2935.
    View in: PubMed
    Score: 0.037
  2. Wang D, Stevens G, Flotte TR. Gene therapy then and now: A look back at changes in the field over the past 25 years. Mol Ther. 2025 May 07; 33(5):1889-1902.
    View in: PubMed
    Score: 0.036
  3. Daci R, Gray-Edwards H, Shazeeb MS, Vardar Z, Vachha B, Cataltepe OI, Flotte TR. Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases. Hum Gene Ther. 2024 Nov; 35(21-22):886-895.
    View in: PubMed
    Score: 0.035
  4. Flotte TR. Intrathecal gene therapy for neurologic disease in humans. Mol Ther. 2024 May 01; 32(5):1185-1186.
    View in: PubMed
    Score: 0.034
  5. Daci R, Flotte TR. Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders. Int J Mol Sci. 2024 Jan 15; 25(2).
    View in: PubMed
    Score: 0.033
  6. Gruntman AM, Xue W, Flotte TR. Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2024; 2750:1-7.
    View in: PubMed
    Score: 0.033
  7. Gruntman AM, Xue W, Flotte TR. Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2024; 2750:11-17.
    View in: PubMed
    Score: 0.033
  8. Lek A, Keeler A, Flotte TR. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. Reply. N Engl J Med. 2023 12 07; 389(23):2211.
    View in: PubMed
    Score: 0.033
  9. Lek A, Wong B, Keeler A, Blackwood M, Ma K, Huang S, Sylvia K, Batista AR, Artinian R, Kokoski D, Parajuli S, Putra J, Carreon CK, Lidov H, Woodman K, Pajusalu S, Spinazzola JM, Gallagher T, LaRovere J, Balderson D, Black L, Sutton K, Horgan R, Lek M, Flotte T. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. N Engl J Med. 2023 09 28; 389(13):1203-1210.
    View in: PubMed
    Score: 0.032
  10. Pires Ferreira D, Gruntman AM, Flotte TR. Gene therapy for alpha-1 antitrypsin deficiency: an update. Expert Opin Biol Ther. 2023 03; 23(3):283-291.
    View in: PubMed
    Score: 0.031
  11. Flotte TR. ESGCT 2022: The New Normal for the Gene Therapy Community. Hum Gene Ther. 2022 09; 33(17-18):841.
    View in: PubMed
    Score: 0.030
  12. Flotte TR, Gessler DJ. Gene Therapy for Rare Neurological Disorders. Clin Pharmacol Ther. 2022 04; 111(4):743-757.
    View in: PubMed
    Score: 0.029
  13. Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH, Martin DR, Gray-Edwards HL, Sena-Esteves M. AAV gene therapy for Tay-Sachs disease. Nat Med. 2022 02; 28(2):251-259.
    View in: PubMed
    Score: 0.029
  14. Flotte TR. Precision of Exon Skipping with U7 Constructs. Hum Gene Ther. 2021 11; 32(21-22):1315-1316.
    View in: PubMed
    Score: 0.028
  15. Batista AR, Flotte TR. In?vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis. Mol Ther. 2021 09 01; 29(9):2633-2634.
    View in: PubMed
    Score: 0.028
  16. Flotte TR, Gao G. Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword. Hum Gene Ther. 2021 07; 32(13-14):645-646.
    View in: PubMed
    Score: 0.028
  17. Flotte TR, Larkin AC, Fischer MA, Chimienti SN, DeMarco DM, Fan PY, Collins MF. In Reply to Ramotshwana et al. Acad Med. 2021 05 01; 96(5):e15-e16.
    View in: PubMed
    Score: 0.027
  18. Flotte TR. Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia. Hum Gene Ther. 2021 May; 32(9-10):449-450.
    View in: PubMed
    Score: 0.027
  19. Flotte TR, Gao G. It Is Time to Call Anti-Asian Bias What It Is: Racism. Hum Gene Ther. 2021 May; 32(9-10):431-432.
    View in: PubMed
    Score: 0.027
  20. Flotte TR, Lord BT, Siedman J. Supporting Families Considering Participation in a Clinical Trial: Parent-Provider Perspectives. Pediatrics. 2021 05; 147(5).
    View in: PubMed
    Score: 0.027
  21. Adair JE, Rubinger KS, Flotte TR. Women Lead Gene Therapy Science in 2021. Hum Gene Ther. 2021 04; 32(7-8):317-318.
    View in: PubMed
    Score: 0.027
  22. Flotte TR. Science Policy and Funding Implications of the 2020 U.S. Election. Hum Gene Ther. 2020 12; 31(23-24):1215-1216.
    View in: PubMed
    Score: 0.027
  23. Flotte TR. Writing the Story of Gene Editing and CRISPR: An Interview with Kevin Davies, PhD. Hum Gene Ther. 2020 12; 31(23-24):1217-1220.
    View in: PubMed
    Score: 0.027
  24. Flotte TR, Larkin AC, Fischer MA, Chimienti SN, DeMarco DM, Fan PY, Collins MF. Accelerated Graduation and the Deployment of New Physicians During the COVID-19 Pandemic. Acad Med. 2020 10; 95(10):1492-1494.
    View in: PubMed
    Score: 0.026
  25. Wilson JM, Flotte TR. Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy. Hum Gene Ther. 2020 07; 31(13-14):695-696.
    View in: PubMed
    Score: 0.026
  26. Flotte TR, Adair JE. Black Lives Matter to Gene Therapy. Hum Gene Ther. 2020 07; 31(13-14):700.
    View in: PubMed
    Score: 0.026
  27. Flotte TR. Barrie J. Carter: The Science and the Scientist. Hum Gene Ther. 2020 05; 31(9-10):489-490.
    View in: PubMed
    Score: 0.026
  28. Flotte TR. Revisiting the "New" Inflammatory Toxicities of Adeno-Associated Virus Vectors. Hum Gene Ther. 2020 04; 31(7-8):398-399.
    View in: PubMed
    Score: 0.025
  29. Flotte TR. Impact on Women's Health: Gene Therapy in Gynecologic Oncology. Hum Gene Ther. 2020 03; 31(5-6):271-272.
    View in: PubMed
    Score: 0.025
  30. Flotte TR. Gene Therapy Untangles the Problem of Chronic Traumatic Encephalopathy. Hum Gene Ther. 2020 01; 31(1-2):12-13.
    View in: PubMed
    Score: 0.025
  31. Flotte TR, Gao G. Prime Editing: A Novel Cas9-Reverse Transcriptase Fusion May Revolutionize Genome Editing. Hum Gene Ther. 2019 12; 30(12):1445-1446.
    View in: PubMed
    Score: 0.025
  32. Flotte TR. Epigenome Editing Strategies for Low Back Pain. Hum Gene Ther. 2019 09; 30(9):1037-1038.
    View in: PubMed
    Score: 0.024
  33. Flotte TR. Getting Tough on Capsid Screening: Tough Decoys Enable Barcoding of Vectors Capable of both Entry and Expression. Hum Gene Ther. 2019 08; 30(8):919-920.
    View in: PubMed
    Score: 0.024
  34. Keeler AM, Flotte TR. Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? Annu Rev Virol. 2019 09 29; 6(1):601-621.
    View in: PubMed
    Score: 0.024
  35. Flotte TR. Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing. Hum Gene Ther. 2019 07; 30(7):793.
    View in: PubMed
    Score: 0.024
  36. Barzel A, Flotte TR. New Horizons for Immune Gene Therapy. Hum Gene Ther. 2019 04; 30(4):379-380.
    View in: PubMed
    Score: 0.024
  37. Flotte TR. Top Five Gene Therapy Stories of 2019. Hum Gene Ther. 2019 01; 30(1):1-2.
    View in: PubMed
    Score: 0.023
  38. Flotte TR. The Year in Review: The Top Five Papers of 2018. Hum Gene Ther. 2018 12; 29(12):1339-1340.
    View in: PubMed
    Score: 0.023
  39. Flotte TR, Lu S. DNA Vaccination in 2018: An Update. Hum Gene Ther. 2018 09; 29(9):963-965.
    View in: PubMed
    Score: 0.023
  40. Flotte TR. Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing. Hum Gene Ther. 2018 08; 29(8):843-844.
    View in: PubMed
    Score: 0.023
  41. Flotte TR. Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy. Hum Gene Ther. 2018 06; 29(6):641-642.
    View in: PubMed
    Score: 0.022
  42. Flotte TR. Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach. Hum Gene Ther. 2018 04; 29(4):401-402.
    View in: PubMed
    Score: 0.022
  43. Gruntman AM, Flotte TR. The rapidly evolving state of gene therapy. FASEB J. 2018 04; 32(4):1733-1740.
    View in: PubMed
    Score: 0.022
  44. Bates CK, Jagsi R, Gordon LK, Travis E, Chatterjee A, Gillis M, Means O, Chaudron L, Ganetzky R, Gulati M, Fivush B, Sharma P, Grover A, Lautenberger D, Flotte TR. It Is Time for Zero Tolerance for Sexual Harassment in Academic Medicine. Acad Med. 2018 02; 93(2):163-165.
    View in: PubMed
    Score: 0.022
  45. Flotte TR, Gao ??? G. Gene and Cell Therapy in China: Highlighting Excellence in the 21st Century : 21. Hum Gene Ther. 2018 02; 29(2):97.
    View in: PubMed
    Score: 0.022
  46. Flotte TR. Therapeutic Advances in Germany and Beyond. Hum Gene Ther. 2017 12; 28(12):1117.
    View in: PubMed
    Score: 0.022
  47. Flotte TR, Daniels E, Benson J, Bevett-Rose JM, Cornetta K, Diggins M, Johnston J, Sepelak S, van der Loo JCM, Wilson JM, McDonald CL. The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute. Hum Gene Ther Clin Dev. 2017 12; 28(4):178-186.
    View in: PubMed
    Score: 0.022
  48. Flotte TR. European Society of Gene and Cell Therapy (ESGCT) at 25: A Gene Therapy Community at Its Prime and on the Move. Hum Gene Ther. 2017 11; 28(11):940.
    View in: PubMed
    Score: 0.022
  49. Flotte TR. Gene Therapy for Alcoholism and Other Substance Use Disorders. Hum Gene Ther. 2017 09; 28(9):701-702.
    View in: PubMed
    Score: 0.021
  50. Flotte TR. Adeno-Associated Virus-Human Bocavirus 1 Chimeric Vectors: Ferreting Out Their Role in Airway Gene Therapy. Hum Gene Ther. 2017 08; 28(8):611.
    View in: PubMed
    Score: 0.021
  51. Keeler AM, ElMallah MK, Flotte TR. Gene Therapy 2017: Progress and Future Directions. Clin Transl Sci. 2017 07; 10(4):242-248.
    View in: PubMed
    Score: 0.021
  52. Mueller C, Gernoux G, Gruntman AM, Borel F, Reeves EP, Calcedo R, Rouhani FN, Yachnis A, Humphries M, Campbell-Thompson M, Messina L, Chulay JD, Trapnell B, Wilson JM, McElvaney NG, Flotte TR. 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency. Mol Ther. 2017 06 07; 25(6):1387-1394.
    View in: PubMed
    Score: 0.021
  53. Wilkins O, Keeler AM, Flotte TR. CAR T-Cell Therapy: Progress and Prospects. Hum Gene Ther Methods. 2017 04; 28(2):61-66.
    View in: PubMed
    Score: 0.021
  54. Flotte TR, Gao G. AAV Is Now a Medicine: We Had Better Get This Right. Hum Gene Ther. 2017 04; 28(4):307.
    View in: PubMed
    Score: 0.021
  55. Flotte TR. The Negative Effects of Immigration Restrictions on the Gene Therapy Community. Hum Gene Ther. 2017 03; 28(3):227.
    View in: PubMed
    Score: 0.021
  56. Flotte TR. Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy? Hum Gene Ther. 2017 02; 28(2):147-148.
    View in: PubMed
    Score: 0.020
  57. Tang Q, Gruntman AM, Flotte TR. Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA. Methods Mol Biol. 2017; 1639:211-216.
    View in: PubMed
    Score: 0.020
  58. Gruntman AM, Flotte TR. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2017; 1639:267-275.
    View in: PubMed
    Score: 0.020
  59. Flotte TR. The Science Policy Implications of a Trump Presidency. Hum Gene Ther. 2017 01; 28(1):1-2.
    View in: PubMed
    Score: 0.020
  60. Naldini L, Parker GC, Flotte TR. The Renaissance of Gene and Cell Therapy: Florence 2016. Hum Gene Ther. 2016 10; 27(10):727-728.
    View in: PubMed
    Score: 0.020
  61. Flotte TR. Gene Drives: Biological Shield or Ecological Menace? Hum Gene Ther. 2016 08; 27(8):561-2.
    View in: PubMed
    Score: 0.020
  62. Flotte TR. The Target's the Thing. Hum Gene Ther. 2016 07; 27(7):477.
    View in: PubMed
    Score: 0.020
  63. Flotte TR. Why Human Gene Therapy Scientists Should Care About Model Organisms. Hum Gene Ther. 2016 May; 27(5):339-40.
    View in: PubMed
    Score: 0.019
  64. Flotte TR. A Gene Therapy Scientist's Life Well-Lived. Hum Gene Ther. 2016 Apr; 27(4):279.
    View in: PubMed
    Score: 0.019
  65. Loring HS, ElMallah MK, Flotte TR. Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans. Hum Gene Ther Methods. 2016 Apr; 27(2):49-58.
    View in: PubMed
    Score: 0.019
  66. Flotte TR. Special Note from the Editor-in-Chief. Hum Gene Ther. 2016 Feb; 27(2):95.
    View in: PubMed
    Score: 0.019
  67. Flotte TR. The Role of Patient Advocacy Organizations in Advancing Human Gene Therapy. Hum Gene Ther. 2015 Dec; 26(12):782.
    View in: PubMed
    Score: 0.019
  68. Flotte TR. Transcription Activator-Like Nucleases Enable Allogeneic Chimeric Antigen Receptor-T Cell Therapy in Humans. Hum Gene Ther. 2015 Nov; 26(11):iv.
    View in: PubMed
    Score: 0.019
  69. Gruntman AM, Flotte TR. Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods. Hum Gene Ther Clin Dev. 2015 Sep; 26(3):159-64.
    View in: PubMed
    Score: 0.019
  70. Flotte TR. Ethical Implications of the Cost of Molecularly Targeted Therapies. Hum Gene Ther. 2015 Sep; 26(9):573-4.
    View in: PubMed
    Score: 0.019
  71. Gruntman AM, Flotte TR. Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency. Hum Gene Ther Methods. 2015 Jun; 26(3):77-81.
    View in: PubMed
    Score: 0.018
  72. Flotte TR. Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question? Hum Gene Ther. 2015 May; 26(5):245-6.
    View in: PubMed
    Score: 0.018
  73. Gruntman AM, Su L, Su Q, Gao G, Mueller C, Flotte TR. Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials. Hum Gene Ther Methods. 2015 Apr; 26(2):71-6.
    View in: PubMed
    Score: 0.018
  74. Loring HS, Flotte TR. Current status of gene therapy for a-1 antitrypsin deficiency. Expert Opin Biol Ther. 2015 Mar; 15(3):329-36.
    View in: PubMed
    Score: 0.017
  75. Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther. 2014 Jul; 22(7):1235-1238.
    View in: PubMed
    Score: 0.017
  76. Flotte TR, Mueller C. What is suppression of anti-adeno-associated virus capsid T-cells achieving? Hum Gene Ther. 2014 Mar; 25(3):178-9.
    View in: PubMed
    Score: 0.017
  77. Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, Sandhaus RA, McElvaney NG, Messina L, Tang Q, Rouhani FN, Campbell-Thompson M, Fu AD, Yachnis A, Knop DR, Ye GJ, Brantly M, Calcedo R, Somanathan S, Richman LP, Vonderheide RH, Hulme MA, Brusko TM, Wilson JM, Flotte TR. Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J Clin Invest. 2013 Dec; 123(12):5310-8.
    View in: PubMed
    Score: 0.016
  78. Flotte TR. Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy. Mol Ther. 2013 Nov; 21(11):1976-81.
    View in: PubMed
    Score: 0.016
  79. Mueller C, Flotte TR. Gene-based therapy for alpha-1 antitrypsin deficiency. COPD. 2013 Mar; 10 Suppl 1:44-9.
    View in: PubMed
    Score: 0.016
  80. Keeler AM, Flotte TR. Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome. Hum Gene Ther. 2012 Jun; 23(6):548-56.
    View in: PubMed
    Score: 0.015
  81. Mueller C, Tang Q, Gruntman A, Blomenkamp K, Teckman J, Song L, Zamore PD, Flotte TR. Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles. Mol Ther. 2012 03; 20(3):590-600.
    View in: PubMed
    Score: 0.014
  82. Ettinger WH, Flotte TR. The role of gene and cell therapy in the era of health care reform. Hum Gene Ther. 2011 Nov; 22(11):1307-9.
    View in: PubMed
    Score: 0.014
  83. Flotte TR, Trapnell BC, Humphries M, Carey B, Calcedo R, Rouhani F, Campbell-Thompson M, Yachnis AT, Sandhaus RA, McElvaney NG, Mueller C, Messina LM, Wilson JM, Brantly M, Knop DR, Ye GJ, Chulay JD. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing a1-antitrypsin: interim results. Hum Gene Ther. 2011 Oct; 22(10):1239-47.
    View in: PubMed
    Score: 0.014
  84. Flotte TR, Mueller C. Gene therapy for alpha-1 antitrypsin deficiency. Hum Mol Genet. 2011 Apr 15; 20(R1):R87-92.
    View in: PubMed
    Score: 0.014
  85. Silver JN, Elder M, Conlon T, Cruz P, Wright AJ, Srivastava A, Flotte TR. Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency. Hum Gene Ther. 2011 Aug; 22(8):935-49.
    View in: PubMed
    Score: 0.014
  86. Flotte TR, Conlon TJ, Mueller C. Preclinical study design for rAAV. Methods Mol Biol. 2011; 807:317-37.
    View in: PubMed
    Score: 0.013
  87. Chulay JD, Ye GJ, Thomas DL, Knop DR, Benson JM, Hutt JA, Wang G, Humphries M, Flotte TR. Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther. 2011 Feb; 22(2):155-65.
    View in: PubMed
    Score: 0.013
  88. Moore MJ, Flotte TR. Autoimmunity in a genetic disease?a cautionary tale. N Engl J Med. 2010 Oct 07; 363(15):1473-5.
    View in: PubMed
    Score: 0.013
  89. Martino AT, Mueller C, Braag S, Cruz PE, Campbell-Thompson M, Jin S, Flotte TR. N-glycosylation augmentation of the cystic fibrosis epithelium improves Pseudomonas aeruginosa clearance. Am J Respir Cell Mol Biol. 2011 Jun; 44(6):824-30.
    View in: PubMed
    Score: 0.013
  90. Flotte TR, Fischer AC, Goetzmann J, Mueller C, Cebotaru L, Yan Z, Wang L, Wilson JM, Guggino WB, Engelhardt JF. Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway. Mol Ther. 2010 Mar; 18(3):594-600.
    View in: PubMed
    Score: 0.012
  91. Stalvey MS, Flotte TR. Endocrine parameters of cystic fibrosis: back to basics. J Cell Biochem. 2009 Oct 01; 108(2):353-61.
    View in: PubMed
    Score: 0.012
  92. Brantly ML, Chulay JD, Wang L, Mueller C, Humphries M, Spencer LT, Rouhani F, Conlon TJ, Calcedo R, Betts MR, Spencer C, Byrne BJ, Wilson JM, Flotte TR. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci U S A. 2009 Sep 22; 106(38):16363-8.
    View in: PubMed
    Score: 0.012
  93. Mueller C, Flotte TR. Gene therapy for cystic fibrosis. Clin Rev Allergy Immunol. 2008 Dec; 35(3):164-78.
    View in: PubMed
    Score: 0.012
  94. Silver JN, Flotte TR. Towards a rAAV-based gene therapy for ADA-SCID: from ADA deficiency to current and future treatment strategies. Pharmacogenomics. 2008 Jul; 9(7):947-68.
    View in: PubMed
    Score: 0.011
  95. Flotte TR. In utero efficacy of cystic fibrosis gene therapy: difficult studies, positive or negative. Mol Ther. 2008 May; 16(5):806-7.
    View in: PubMed
    Score: 0.011
  96. Mueller C, Flotte TR. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther. 2008 Jun; 15(11):858-63.
    View in: PubMed
    Score: 0.011
  97. Mueller C, Torrez D, Braag S, Martino A, Clarke T, Campbell-Thompson M, Flotte TR. Partial correction of the CFTR-dependent ABPA mouse model with recombinant adeno-associated virus gene transfer of truncated CFTR gene. J Gene Med. 2008 Jan; 10(1):51-60.
    View in: PubMed
    Score: 0.011
  98. Flotte TR. Gene therapy: the first two decades and the current state-of-the-art. J Cell Physiol. 2007 Nov; 213(2):301-5.
    View in: PubMed
    Score: 0.011
  99. Cruz PE, Mueller C, Flotte TR. The promise of gene therapy for the treatment of alpha-1 antitrypsin deficiency. Pharmacogenomics. 2007 Sep; 8(9):1191-8.
    View in: PubMed
    Score: 0.011
  100. Cruz PE, Mueller C, Cossette TL, Golant A, Tang Q, Beattie SG, Brantly M, Campbell-Thompson M, Blomenkamp KS, Teckman JH, Flotte TR. In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. Lab Invest. 2007 Sep; 87(9):893-902.
    View in: PubMed
    Score: 0.010
  101. Erger KE, Conlon TJ, Leal NA, Zori R, Bobik TA, Flotte TR. In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8. J Gene Med. 2007 Jun; 9(6):462-9.
    View in: PubMed
    Score: 0.010
  102. Flotte TR, Conlon TJ, Poirier A, Campbell-Thompson M, Byrne BJ. Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites. Hum Gene Ther. 2007 Mar; 18(3):245-56.
    View in: PubMed
    Score: 0.010
  103. Flotte TR, Ng P, Dylla DE, McCray PB, Wang G, Kolls JK, Hu J. Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol Ther. 2007 Feb; 15(2):229-41.
    View in: PubMed
    Score: 0.010
  104. Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther. 2006 Dec; 17(12):1177-86.
    View in: PubMed
    Score: 0.010
  105. Flotte TR, Frentzen B, Humphries MR, Rosenbloom AL. Recent developments in the protection of pediatric research subjects. J Pediatr. 2006 Sep; 149(3):285-286.
    View in: PubMed
    Score: 0.010
  106. Conlon TJ, Walter G, Owen R, Cossette T, Erger K, Gutierrez G, Goetzman E, Matern D, Vockley J, Flotte TR. Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector. Hum Gene Ther. 2006 Jan; 17(1):71-80.
    View in: PubMed
    Score: 0.009
  107. Kaplan KA, Beierle EA, Faro A, Eskin TA, Flotte TR. Recurrent pneumonia in children: a case report and approach to diagnosis. Clin Pediatr (Phila). 2006 Jan-Feb; 45(1):15-22.
    View in: PubMed
    Score: 0.009
  108. Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res. 2005 Dec; 58(6):1143-7.
    View in: PubMed
    Score: 0.009
  109. Xing R, Xu M, Reil D, Destefano A, Cui M, Liu N, Liang J, Xu G, Luo L, Xu M, Zhang F, Tai PWL, Wei Y, Gruntman AM, Flotte TR, Gao G, Wang D. A single amino acid variant in the variable region I of AAV capsid confers liver detargeting. PLoS Pathog. 2025 Sep; 21(9):e1013533.
    View in: PubMed
    Score: 0.009
  110. Conlon TJ, Cossette T, Erger K, Choi YK, Clarke T, Scott-Jorgensen M, Song S, Campbell-Thompson M, Crawford J, Flotte TR. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. Mol Ther. 2005 Nov; 12(5):867-75.
    View in: PubMed
    Score: 0.009
  111. Flotte TR, Schwiebert EM, Zeitlin PL, Carter BJ, Guggino WB. Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy. Hum Gene Ther. 2005 Aug; 16(8):921-8.
    View in: PubMed
    Score: 0.009
  112. Virella-Lowell I, Zusman B, Foust K, Loiler S, Conlon T, Song S, Chesnut KA, Ferkol T, Flotte TR. Enhancing rAAV vector expression in the lung. J Gene Med. 2005 Jul; 7(7):842-50.
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  113. Flotte TR. Adeno-associated virus-mediated gene transfer for lung diseases. Hum Gene Ther. 2005 Jun; 16(6):643-8.
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  114. Flotte TR. Recent developments in recombinant AAV-mediated gene therapy for lung diseases. Curr Gene Ther. 2005 Jun; 5(3):361-6.
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  115. Flotte TR, Berns KI. Adeno-associated virus: a ubiquitous commensal of mammals. Hum Gene Ther. 2005 Apr; 16(4):401-7.
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  116. Pires-Ferreira D, Reil D, Tang Q, Blackwood M, Gallagher T, Keeler AM, Chichester JA, Vyhnal KK, Lindborg JA, Benson J, Fu D, Flotte TR, Gruntman AM. Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy. Genes (Basel). 2024 Sep 10; 15(9).
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  117. Virella-Lowell I, Herlihy JD, Liu B, Lopez C, Cruz P, Muller C, Baker HV, Flotte TR. Effects of CFTR, interleukin-10, and Pseudomonas aeruginosa on gene expression profiles in a CF bronchial epithelial cell Line. Mol Ther. 2004 Sep; 10(3):562-73.
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    Score: 0.009
  118. Liang SQ, Navia AW, Ramseier M, Zhou X, Martinez M, Lee C, Zhou C, Wu J, Xie J, Su Q, Wang D, Flotte TR, Anderson DG, Tarantal AF, Shalek AK, Gao G, Xue W. AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys. Hum Gene Ther. 2024 10; 35(19-20):814-824.
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    Score: 0.009
  119. Flotte TR. Immune responses to recombinant adeno-associated virus vectors: putting preclinical findings into perspective. Hum Gene Ther. 2004 Jul; 15(7):716-7.
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  120. Conlon TJ, Flotte TR. Recombinant adeno-associated virus vectors for gene therapy. Expert Opin Biol Ther. 2004 Jul; 4(7):1093-101.
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  121. Flotte TR. Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2004 May; 11(10):805-10.
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  122. Song S, Lu Y, Choi YK, Han Y, Tang Q, Zhao G, Berns KI, Flotte TR. DNA-dependent PK inhibits adeno-associated virus DNA integration. Proc Natl Acad Sci U S A. 2004 Feb 17; 101(7):2112-6.
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    Score: 0.008
  123. Song S, Goudy K, Campbell-Thompson M, Wasserfall C, Scott-Jorgensen M, Wang J, Tang Q, Crawford JM, Ellis TM, Atkinson MA, Flotte TR. Recombinant adeno-associated virus-mediated alpha-1 antitrypsin gene therapy prevents type I diabetes in NOD mice. Gene Ther. 2004 Jan; 11(2):181-6.
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    Score: 0.008
  124. Flotte TR, Brantly ML, Spencer LT, Byrne BJ, Spencer CT, Baker DJ, Humphries M. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther. 2004 Jan; 15(1):93-128.
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    Score: 0.008
  125. Loiler SA, Conlon TJ, Song S, Tang Q, Warrington KH, Agarwal A, Kapturczak M, Li C, Ricordi C, Atkinson MA, Muzyczka N, Flotte TR. Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver. Gene Ther. 2003 Sep; 10(18):1551-8.
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  126. Flotte TR, Zeitlin PL, Reynolds TC, Heald AE, Pedersen P, Beck S, Conrad CK, Brass-Ernst L, Humphries M, Sullivan K, Wetzel R, Taylor G, Carter BJ, Guggino WB. Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum Gene Ther. 2003 Jul 20; 14(11):1079-88.
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  127. Stacpoole PW, Owen R, Flotte TR. The pyruvate dehydrogenase complex as a target for gene therapy. Curr Gene Ther. 2003 Jun; 3(3):239-45.
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  128. Flotte TR. The signal and the trap: targeted delivery and retention of proteins in the mitochondrion. Mol Ther. 2003 Jun; 7(6):715-6.
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  129. Arjomandnejad M, Dasgupta I, Flotte TR, Keeler AM. Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer. BioDrugs. 2023 May; 37(3):311-329.
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  130. Milner RJ, Flotte TR, Thorndyke LE. Defining Scholarship for Today and Tomorrow. J Contin Educ Health Prof. 2023 04 01; 43(2):133-138.
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  131. Snyder RO, Flotte TR. Production of clinical-grade recombinant adeno-associated virus vectors. Curr Opin Biotechnol. 2002 Oct; 13(5):418-23.
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  132. Owen R, Mandel RJ, Ammini CV, Conlon TJ, Kerr DS, Stacpoole PW, Flotte TR. Gene therapy for pyruvate dehydrogenase E1alpha deficiency using recombinant adeno-associated virus 2 (rAAV2) vectors. Mol Ther. 2002 Sep; 6(3):394-9.
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  133. Ke H, Guay KP, Flotte TR, Gierasch LM, Gershenson A, Hebert DN. Secretion of functional a1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy. Proc Natl Acad Sci U S A. 2022 08 02; 119(31):e2206103119.
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  134. Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, B?nnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M. Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders. Mol Ther. 2022 07 06; 30(7):2416-2428.
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  135. Flotte TR. Recombinant adeno-associated virus gene therapy for cystic fibrosis and alpha(1)-antitrypsin deficiency. Chest. 2002 Mar; 121(3 Suppl):98S-102S.
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  136. Flotte TR, Virella-Lowell I, Chesnut KA. Adeno-associated viral vectors for CF gene therapy. Methods Mol Med. 2002; 70:599-608.
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  137. Mah C, Byrne BJ, Flotte TR. Virus-based gene delivery systems. Clin Pharmacokinet. 2002; 41(12):901-11.
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  138. Flotte TR. Recombinant adeno-associated virus vectors for cystic fibrosis gene therapy. Curr Opin Mol Ther. 2001 Oct; 3(5):497-502.
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  139. Flotte TR, Laube BL. Gene therapy in cystic fibrosis. Chest. 2001 Sep; 120(3 Suppl):124S-131S.
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  140. Song S, Embury J, Laipis PJ, Berns KI, Crawford JM, Flotte TR. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2001 Sep; 8(17):1299-306.
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  141. Owen R, Flotte TR. Approaches and limitations to gene therapy for mitochondrial diseases. Antioxid Redox Signal. 2001 Jun; 3(3):451-60.
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  142. Liu P, Liang SQ, Zheng C, Mintzer E, Zhao YG, Ponnienselvan K, Mir A, Sontheimer EJ, Gao G, Flotte TR, Wolfe SA, Xue W. Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice. Nat Commun. 2021 04 09; 12(1):2121.
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  143. Qin W, Xu G, Tai PWL, Wang C, Luo L, Li C, Hu X, Xue J, Lu Y, Zhou Q, Wei Q, Wen T, Hu J, Xiao Y, Yang L, Li W, Flotte TR, Wei Y, Gao G. Large-scale molecular epidemiological analysis of AAV in a cancer patient population. Oncogene. 2021 Apr; 40(17):3060-3071.
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    Score: 0.007
  144. Jacobson CE, Beeler WH, Griffith KA, Flotte TR, Byington CL, Jagsi R. Common pathways to Dean of Medicine at U.S. medical schools. PLoS One. 2021; 16(3):e0249078.
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  145. Flotte T, Agarwal A, Wang J, Song S, Fenjves ES, Inverardi L, Chesnut K, Afione S, Loiler S, Wasserfall C, Kapturczak M, Ellis T, Nick H, Atkinson M. Efficient ex vivo transduction of pancreatic islet cells with recombinant adeno-associated virus vectors. Diabetes. 2001 Mar; 50(3):515-20.
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  146. Dasgupta I, Flotte TR, Keeler AM. CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing. Hum Gene Ther. 2021 03; 32(5-6):275-293.
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    Score: 0.007
  147. B?ning H, Baker AH, Griesenbach U, Flotte TR, Thrasher AJ. Gene and Cell Therapy for Inherited and Acquired Immune Deficiency. Hum Gene Ther. 2021 01; 32(1-2):1-3.
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    Score: 0.007
  148. Fishbein L, O'Brien P, Hutson A, Theriaque D, Stacpoole PW, Flotte T. Pharmacokinetics and pharmacodynamic effects of nicotine nasal spray devices on cardiovascular and pulmonary function. J Investig Med. 2000 Nov; 48(6):435-40.
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  149. Owen R IV, Lewin AP, Peel A, Wang J, Guy J, Hauswirth WW, Stacpoole PW, Flotte TR. Recombinant adeno-associated virus vector-based gene transfer for defects in oxidative metabolism. Hum Gene Ther. 2000 Oct 10; 11(15):2067-78.
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  150. Virella-Lowell I, Poirier A, Chesnut KA, Brantly M, Flotte TR. Inhibition of recombinant adeno-associated virus (rAAV) transduction by bronchial secretions from cystic fibrosis patients. Gene Ther. 2000 Oct; 7(20):1783-9.
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  151. Jagsi R, Means O, Lautenberger D, Jones RD, Griffith KA, Flotte TR, Gordon LK, Rexrode KM, Wagner LW, Chatterjee A. Women's Representation Among Members and Leaders of National Medical Specialty Societies. Acad Med. 2020 07; 95(7):1043-1049.
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    Score: 0.006
  152. Taghian T, Horn E, Shazeeb MS, Bierfeldt LJ, Tuominen SM, Koehler J, Fernau D, Bertrand S, Frey S, Cataltepe OI, Gounis MJ, Abayazeed AH, Flotte TR, Sena-Esteves M, Gray-Edwards HL. Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model. Hum Gene Ther. 2020 06; 31(11-12):617-625.
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  153. Gernoux G, Gruntman AM, Blackwood M, Zieger M, Flotte TR, Mueller C. Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans. Mol Ther. 2020 03 04; 28(3):747-757.
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  154. Tang Q, Gernoux G, Cheng Y, Flotte T, Mueller C. Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model. Methods Mol Biol. 2020; 2164:75-85.
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    Score: 0.006
  155. Taghian T, Marosfoi MG, Puri AS, Cataltepe OI, King RM, Diffie EB, Maguire AS, Martin DR, Fernau D, Batista AR, Kuchel T, Christou C, Perumal R, Chandra S, Gamlin PD, Bertrand SG, Flotte TR, McKenna-Yasek D, Tai PWL, Aronin N, Gounis MJ, Sena-Esteves M, Gray-Edwards HL. A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna. Mol Ther. 2020 02 05; 28(2):411-421.
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  156. Flotte TR. Gene therapy for cystic fibrosis. Curr Opin Mol Ther. 1999 Aug; 1(4):510-6.
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  157. Chimienti SN, DeMarco DM, Flotte TR, Collins MF. Assuring Integrity in the Residency Match Process. Acad Med. 2019 03; 94(3):321-323.
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  158. Afione SA, Wang J, Walsh S, Guggino WB, Flotte TR. Delayed expression of adeno-associated virus vector DNA. Intervirology. 1999; 42(4):213-20.
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  159. Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A. 1998 Nov 24; 95(24):14384-8.
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  160. Pennesi ME, Weleber RG, Yang P, Whitebirch C, Thean B, Flotte TR, Humphries M, Chegarnov E, Beasley KN, Stout JT, Chulay JD. Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy. Hum Gene Ther. 2018 12; 29(12):1428-1437.
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    Score: 0.006
  161. Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. Hum Gene Ther. 2018 08; 29(8):853-860.
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  162. Borel F, Sun H, Zieger M, Cox A, Cardozo B, Li W, Oliveira G, Davis A, Gruntman A, Flotte TR, Brodsky MH, Hoffman AM, Elmallah MK, Mueller C. Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema. Proc Natl Acad Sci U S A. 2018 03 13; 115(11):2788-2793.
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  163. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol. 1998; 292:717-32.
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  164. Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C. Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of a-1 Antitrypsin Deficiency. Mol Ther. 2017 11 01; 25(11):2477-2489.
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  165. Stoica L, Keeler AM, Xiong L, Kalfopoulos M, Desrochers K, Brown RH, Sena-Esteves M, Flotte TR, ElMallah MK. Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model. Am J Respir Cell Mol Biol. 2017 03; 56(3):405-408.
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  166. Flotte TR, Ferkol TW. Genetic therapy. Past, present, and future. Pediatr Clin North Am. 1997 Feb; 44(1):153-78.
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  167. Calcedo R, Somanathan S, Qin Q, Betts MR, Rech AJ, Vonderheide RH, Mueller C, Flotte TR, Wilson JM. Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for a-1-antitrypsin deficiency. Proc Natl Acad Sci U S A. 2017 02 14; 114(7):1655-1659.
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  168. Flotte TR, Carter BJ. In vivo gene therapy with adeno-associated virus vectors for cystic fibrosis. Adv Pharmacol. 1997; 40:85-101.
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  169. Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, Rosenstein B, Taylor G, Walden S, Wetzel R. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther. 1996 Jun 10; 7(9):1145-59.
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  170. Afione SA, Conrad CK, Kearns WG, Chunduru S, Adams R, Reynolds TC, Guggino WB, Cutting GR, Carter BJ, Flotte TR. In vivo model of adeno-associated virus vector persistence and rescue. J Virol. 1996 May; 70(5):3235-41.
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  171. Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy. Ophthalmology. 2016 07; 123(7):1606-20.
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  172. Agre P, Bertozzi C, Bissell M, Campbell KP, Cummings RD, Desai UR, Estes M, Flotte T, Fogleman G, Gage F, Ginsburg D, Gordon JI, Hart G, Hascall V, Kiessling L, Kornfeld S, Lowe J, Magnani J, Mahal LK, Medzhitov R, Roberts RJ, Sackstein R, Sarkar R, Schnaar R, Schwartz N, Varki A, Walt D, Weissman I. Training the next generation of biomedical investigators in glycosciences. J Clin Invest. 2016 Feb; 126(2):405-8.
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  173. Carter BJ, Flotte TR. Development of adeno-associated virus vectors for gene therapy of cystic fibrosis. Curr Top Microbiol Immunol. 1996; 218:119-44.
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  174. Schnepp BC, Chulay JD, Ye GJ, Flotte TR, Trapnell BC, Johnson PR. Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle. Hum Gene Ther. 2016 Jan; 27(1):32-42.
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  175. Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A. Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? Hum Gene Ther. 2015 Dec; 26(12):779-81.
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  176. Li S, Ling C, Zhong L, Li M, Su Q, He R, Tang Q, Greiner DL, Shultz LD, Brehm MA, Flotte TR, Mueller C, Srivastava A, Gao G. Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors. Mol Ther. 2015 Dec; 23(12):1867-76.
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  177. Flotte TR, Carter BJ. Adeno-associated virus vectors for gene therapy. Gene Ther. 1995 Aug; 2(6):357-62.
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  178. Afione SA, Conrad CK, Flotte TR. Gene therapy vectors as drug delivery systems. Clin Pharmacokinet. 1995 Mar; 28(3):181-9.
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  179. Flotte TR, Afione SA, Zeitlin PL. Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol. 1994 Nov; 11(5):517-21.
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  180. Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci U S A. 1993 Nov 15; 90(22):10613-7.
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  181. Ahmed SS, Li H, Cao C, Sikoglu EM, Denninger AR, Su Q, Eaton S, Liso Navarro AA, Xie J, Szucs S, Zhang H, Moore C, Kirschner DA, Seyfried TN, Flotte TR, Matalon R, Gao G. A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice. Mol Ther. 2013 Dec; 21(12):2136-47.
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  182. Flotte TR, Afione SA, Solow R, Drumm ML, Markakis D, Guggino WB, Zeitlin PL, Carter BJ. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J Biol Chem. 1993 Feb 15; 268(5):3781-90.
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  183. Flotte TR. Prospects for virus-based gene therapy for cystic fibrosis. J Bioenerg Biomembr. 1993 Feb; 25(1):37-42.
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  184. Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochm?ller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther. 2013 Feb; 21(2):266-8.
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  185. Bouchecareilh M, Hutt DM, Szajner P, Flotte TR, Balch WE. Histone deacetylase inhibitor (HDACi) suberoylanilide hydroxamic acid (SAHA)-mediated correction of a1-antitrypsin deficiency. J Biol Chem. 2012 Nov 02; 287(45):38265-78.
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  186. Bickford JS, Mueller C, Newsom KJ, Barilovits SJ, Beachy DE, Herlihy JD, Keeler B, Flotte TR, Nick HS. Effect of allergy and inflammation on eicosanoid gene expression in CFTR deficiency. J Cyst Fibros. 2013 May; 12(3):258-65.
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  187. Flotte TR, Solow R, Owens RA, Afione S, Zeitlin PL, Carter BJ. Gene expression from adeno-associated virus vectors in airway epithelial cells. Am J Respir Cell Mol Biol. 1992 Sep; 7(3):349-56.
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  188. Lockett AD, Van Demark M, Gu Y, Schweitzer KS, Sigua N, Kamocki K, Fijalkowska I, Garrison J, Fisher AJ, Serban K, Wise RA, Flotte TR, Mueller C, Presson RG, Petrache HI, Tuder RM, Petrache I. Effect of cigarette smoke exposure and structural modifications on the a-1 Antitrypsin interaction with caspases. Mol Med. 2012 May 09; 18:445-54.
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  189. Bickford JS, Newsom KJ, Herlihy JD, Mueller C, Keeler B, Qiu X, Walters JN, Su N, Wallet SM, Flotte TR, Nick HS. Induction of group IVC phospholipase A2 in allergic asthma: transcriptional regulation by TNFa in bronchoepithelial cells. Biochem J. 2012 Feb 15; 442(1):127-37.
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  190. Chen KA, Cruz PE, Lanuto DJ, Flotte TR, Borchelt DR, Srivastava A, Zhang J, Steindler DA, Zheng T. Cellular fusion for gene delivery to SCA1 affected Purkinje neurons. Mol Cell Neurosci. 2011 May; 47(1):61-70.
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  191. Flotte TR, Loughlin GM. Benefits and complications of troleandomycin (TAO) in young children with steroid-dependent asthma. Pediatr Pulmonol. 1991; 10(3):178-82.
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    Score: 0.003
  192. Xie J, Xie Q, Zhang H, Ameres SL, Hung JH, Su Q, He R, Mu X, Seher Ahmed S, Park S, Kato H, Li C, Mueller C, Mello CC, Weng Z, Flotte TR, Zamore PD, Gao G. MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression. Mol Ther. 2011 Mar; 19(3):526-35.
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  193. Goel VK, Ibrahim N, Jiang G, Singhal M, Fee S, Flotte T, Westmoreland S, Haluska FS, Hinds PW, Haluska FG. Melanocytic nevus-like hyperplasia and melanoma in transgenic BRAFV600E mice. Oncogene. 2009 Jun 11; 28(23):2289-98.
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  194. Yu H, Zeidan YH, Wu BX, Jenkins RW, Flotte TR, Hannun YA, Virella-Lowell I. Defective acid sphingomyelinase pathway with Pseudomonas aeruginosa infection in cystic fibrosis. Am J Respir Cell Mol Biol. 2009 Sep; 41(3):367-75.
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  195. Flotte TR. Dietl syndrome: intermittent ureteropelvic junction obstruction as a cause of episodic abdominal pain. Pediatrics. 1988 Nov; 82(5):792-4.
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  196. Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther. 2008 Oct; 19(10):979-90.
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  197. Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ, Pang JJ, Sumaroka A, Windsor EA, Wilson JM, Flotte TR, Fishman GA, Heon E, Stone EM, Byrne BJ, Jacobson SG, Hauswirth WW. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A. 2008 Sep 30; 105(39):15112-7.
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    Score: 0.003
  198. Alexander BL, Ali RR, Alton EW, Bainbridge JW, Braun S, Cheng SH, Flotte TR, Gaspar HB, Grez M, Griesenbach U, Kaplitt MG, Ott MG, Seger R, Simons M, Thrasher AJ, Thrasher AZ, Yl?-Herttuala S. Progress and prospects: gene therapy clinical trials (part 1). Gene Ther. 2007 Oct; 14(20):1439-47.
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    Score: 0.003
  199. Petrache I, Fijalkowska I, Medler TR, Skirball J, Cruz P, Zhen L, Petrache HI, Flotte TR, Tuder RM. alpha-1 antitrypsin inhibits caspase-3 activity, preventing lung endothelial cell apoptosis. Am J Pathol. 2006 Oct; 169(4):1155-66.
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    Score: 0.002
  200. Milligan ED, Sloane EM, Langer SJ, Hughes TS, Jekich BM, Frank MG, Mahoney JH, Levkoff LH, Maier SF, Cruz PE, Flotte TR, Johnson KW, Mahoney MM, Chavez RA, Leinwand LA, Watkins LR. Repeated intrathecal injections of plasmid DNA encoding interleukin-10 produce prolonged reversal of neuropathic pain. Pain. 2006 Dec 15; 126(1-3):294-308.
    View in: PubMed
    Score: 0.002
  201. Jacobson SG, Boye SL, Aleman TS, Conlon TJ, Zeiss CJ, Roman AJ, Cideciyan AV, Schwartz SB, Komaromy AM, Doobrajh M, Cheung AY, Sumaroka A, Pearce-Kelling SE, Aguirre GD, Kaushal S, Maguire AM, Flotte TR, Hauswirth WW. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther. 2006 Aug; 17(8):845-58.
    View in: PubMed
    Score: 0.002
  202. Lu Y, Choi YK, Campbell-Thompson M, Li C, Tang Q, Crawford JM, Flotte TR, Song S. Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector. J Gene Med. 2006 Jun; 8(6):730-5.
    View in: PubMed
    Score: 0.002
  203. Petrache I, Fijalkowska I, Zhen L, Medler TR, Brown E, Cruz P, Choe KH, Taraseviciene-Stewart L, Scerbavicius R, Shapiro L, Zhang B, Song S, Hicklin D, Voelkel NF, Flotte T, Tuder RM. A novel antiapoptotic role for alpha1-antitrypsin in the prevention of pulmonary emphysema. Am J Respir Crit Care Med. 2006 Jun 01; 173(11):1222-8.
    View in: PubMed
    Score: 0.002
  204. Swiatecka-Urban A, Brown A, Moreau-Marquis S, Renuka J, Coutermarsh B, Barnaby R, Karlson KH, Flotte TR, Fukuda M, Langford GM, Stanton BA. The short apical membrane half-life of rescued {Delta}F508-cystic fibrosis transmembrane conductance regulator (CFTR) results from accelerated endocytosis of {Delta}F508-CFTR in polarized human airway epithelial cells. J Biol Chem. 2005 Nov 04; 280(44):36762-72.
    View in: PubMed
    Score: 0.002
  205. Milligan ED, Sloane EM, Langer SJ, Cruz PE, Chacur M, Spataro L, Wieseler-Frank J, Hammack SE, Maier SF, Flotte TR, Forsayeth JR, Leinwand LA, Chavez R, Watkins LR. Controlling neuropathic pain by adeno-associated virus driven production of the anti-inflammatory cytokine, interleukin-10. Mol Pain. 2005 Feb 25; 1:9.
    View in: PubMed
    Score: 0.002
  206. Mohiuddin I, Loiler S, Zolotukhin I, Byrne BJ, Flotte TR, Snyder RO. Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. Mol Ther. 2005 Feb; 11(2):320-6.
    View in: PubMed
    Score: 0.002
  207. Chen S, Kapturczak M, Loiler SA, Zolotukhin S, Glushakova OY, Madsen KM, Samulski RJ, Hauswirth WW, Campbell-Thompson M, Berns KI, Flotte TR, Atkinson MA, Tisher CC, Agarwal A. Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Hum Gene Ther. 2005 Feb; 16(2):235-47.
    View in: PubMed
    Score: 0.002
  208. Bray CL, Cahill KS, Oshier JT, Peden CS, Theriaque DW, Flotte TR, Stacpoole PW. Methylphenidate does not improve cognitive function in healthy sleep-deprived young adults. J Investig Med. 2004 Apr; 52(3):192-201.
    View in: PubMed
    Score: 0.002
  209. Burkhardt BR, Loiler SA, Anderson JA, Kilberg MS, Crawford JM, Flotte TR, Goudy KS, Ellis TM, Atkinson M. Glucose-responsive expression of the human insulin promoter in HepG2 human hepatoma cells. Ann N Y Acad Sci. 2003 Nov; 1005:237-41.
    View in: PubMed
    Score: 0.002
  210. Seo BB, Nakamaru-Ogiso E, Flotte TR, Yagi T, Matsuno-Yagi A. A single-subunit NADH-quinone oxidoreductase renders resistance to mammalian nerve cells against complex I inhibition. Mol Ther. 2002 Sep; 6(3):336-41.
    View in: PubMed
    Score: 0.002
  211. Wagner JA, Nepomuceno IB, Messner AH, Moran ML, Batson EP, Dimiceli S, Brown BW, Desch JK, Norbash AM, Conrad CK, Guggino WB, Flotte TR, Wine JJ, Carter BJ, Reynolds TC, Moss RB, Gardner P. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther. 2002 Jul 20; 13(11):1349-59.
    View in: PubMed
    Score: 0.002
  212. Mah C, Fraites TJ, Zolotukhin I, Song S, Flotte TR, Dobson J, Batich C, Byrne BJ. Improved method of recombinant AAV2 delivery for systemic targeted gene therapy. Mol Ther. 2002 Jul; 6(1):106-12.
    View in: PubMed
    Score: 0.002
  213. Kapturczak M, Zolotukhin S, Cross J, Pileggi A, Molano RD, Jorgensen M, Byrne B, Flotte TR, Ellis T, Inverardi L, Ricordi C, Nick H, Atkinson M, Agarwal A. Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector. Mol Ther. 2002 Feb; 5(2):154-60.
    View in: PubMed
    Score: 0.002
  214. Goudy K, Song S, Wasserfall C, Zhang YC, Kapturczak M, Muir A, Powers M, Scott-Jorgensen M, Campbell-Thompson M, Crawford JM, Ellis TM, Flotte TR, Atkinson MA. Adeno-associated virus vector-mediated IL-10 gene delivery prevents type 1 diabetes in NOD mice. Proc Natl Acad Sci U S A. 2001 Nov 20; 98(24):13913-8.
    View in: PubMed
    Score: 0.002
  215. Kapturczak MH, Flotte T, Atkinson MA. Adeno-associated virus (AAV) as a vehicle for therapeutic gene delivery: improvements in vector design and viral production enhance potential to prolong graft survival in pancreatic islet cell transplantation for the reversal of type 1 diabetes. Curr Mol Med. 2001 May; 1(2):245-58.
    View in: PubMed
    Score: 0.002
  216. Xu L, Daly T, Gao C, Flotte TR, Song S, Byrne BJ, Sands MS, Parker Ponder K. CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice. Hum Gene Ther. 2001 Mar 20; 12(5):563-73.
    View in: PubMed
    Score: 0.002
  217. Stacpoole PW, Fisher WR, Flotte TR, Geiser EA, Theriaque DW, Hutson AD. Teaching hypothesis-oriented thinking to medical students: the University of Florida's clinical investigation program. Acad Med. 2001 Mar; 76(3):287-92.
    View in: PubMed
    Score: 0.002
  218. Seo BB, Wang J, Flotte TR, Yagi T, Matsuno-Yagi A. Use of the NADH-quinone oxidoreductase (NDI1) gene of Saccharomyces cerevisiae as a possible cure for complex I defects in human cells. J Biol Chem. 2000 Dec 01; 275(48):37774-8.
    View in: PubMed
    Score: 0.002
  219. Wu P, Xiao W, Conlon T, Hughes J, Agbandje-McKenna M, Ferkol T, Flotte T, Muzyczka N. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol. 2000 Sep; 74(18):8635-47.
    View in: PubMed
    Score: 0.002
  220. Wagner JA, Messner AH, Moran ML, Daifuku R, Kouyama K, Desch JK, Manley S, Norbash AM, Conrad CK, Friborg S, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P. Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. Laryngoscope. 1999 Feb; 109(2 Pt 1):266-74.
    View in: PubMed
    Score: 0.001
  221. Wagner JA, Reynolds T, Moran ML, Moss RB, Wine JJ, Flotte TR, Gardner P. Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus. Lancet. 1998 Jun 06; 351(9117):1702-3.
    View in: PubMed
    Score: 0.001
  222. Wagner JA, Moran ML, Messner AH, Daifuku R, Conrad CK, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P. A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. Hum Gene Ther. 1998 Apr 10; 9(6):889-909.
    View in: PubMed
    Score: 0.001
  223. Grossman MC, Dierickx C, Farinelli W, Flotte T, Anderson RR. Damage to hair follicles by normal-mode ruby laser pulses. J Am Acad Dermatol. 1996 Dec; 35(6):889-94.
    View in: PubMed
    Score: 0.001
  224. Kearns WG, Afione SA, Fulmer SB, Pang MC, Erikson D, Egan M, Landrum MJ, Flotte TR, Cutting GR. Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther. 1996 Sep; 3(9):748-55.
    View in: PubMed
    Score: 0.001
  225. Baudard M, Flotte TR, Aran JM, Thierry AR, Pastan I, Pang MG, Kearns WG, Gottesman MM. Expression of the human multidrug resistance and glucocerebrosidase cDNAs from adeno-associated vectors: efficient promoter activity of AAV sequences and in vivo delivery via liposomes. Hum Gene Ther. 1996 Jul 10; 7(11):1309-22.
    View in: PubMed
    Score: 0.001
  226. Carroll TP, Morales MM, Fulmer SB, Allen SS, Flotte TR, Cutting GR, Guggino WB. Alternate translation initiation codons can create functional forms of cystic fibrosis transmembrane conductance regulator. J Biol Chem. 1995 May 19; 270(20):11941-6.
    View in: PubMed
    Score: 0.001
  227. Schwiebert EM, Flotte T, Cutting GR, Guggino WB. Both CFTR and outwardly rectifying chloride channels contribute to cAMP-stimulated whole cell chloride currents. Am J Physiol. 1994 May; 266(5 Pt 1):C1464-77.
    View in: PubMed
    Score: 0.001
  228. Tomares SM, Flotte TR, Tunkel DE, Pao M, Loughlin GM. Real time laryngoscopy with olfactory challenge for diagnosis of psychogenic stridor. Pediatr Pulmonol. 1993 Oct; 16(4):259-62.
    View in: PubMed
    Score: 0.001
  229. Egan M, Flotte T, Afione S, Solow R, Zeitlin PL, Carter BJ, Guggino WB. Defective regulation of outwardly rectifying Cl- channels by protein kinase A corrected by insertion of CFTR. Nature. 1992 Aug 13; 358(6387):581-4.
    View in: PubMed
    Score: 0.001
Connection Strength

The connection strength for concepts is the sum of the scores for each matching publication.

Publication scores are based on many factors, including how long ago they were written and whether the person is a first or senior author.